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Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027
Read Avidity’s Community Letter:
STAT: A decade ago, these drugs tore apart the FDA. Today might be some patients’ best hope.
By Jason Mast
Avidity Biosciences Managed Access Program (MAP) – Frequently Asked Questions
Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. […]
Dyne Webinar: Positive Topline Results from DELIVER trial in Exon 51 DMD
A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits […]
Avidity’s New Access Program Marks a Milestone for the Duchenne Community
In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. […]
PepGen ends development of Duchenne Exon-51 skipping program
PepGen has ended clinical trials of PGN-EDO51, their investigational therapy for Duchenne patients amenable to skipping exon 51, as PGN-EDO51 failed to achieve target dystrophin levels in their CONNECT1 trial. The company intends […]
Entrada receives authorization to start exon 44 skipping trial in Europe
CureDuchenne provided early funding to Entrada Therapeutics. Entrada has received regulatory authorization in Europe to initiate a Phase 1/2 clinical trial of ENTR-601-44 in ambulatory individuals with Duchenne amenable to […]
Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study
Wave Life Science Exon Skipping in Duchenne Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study: An update on Wave’s Phase 2 FORWARD-53 trial of WE-N531, Wave’s […]
Dyne Therapeutics: The clinical impact of the FORCE™ platform
Duchenne muscular dystrophy (DMD) – An update on the DELIVER trial of DYNE-251 in males with DMD mutations amenable to exon 51 skipping. WATCH HERE